New hope for rare bleeding disorder: experimental drug tested in small study

NCT ID NCT04770935

Completed Disease control Sponsor: Bioverativ, a Sanofi company Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 31 times

Summary

This early-stage study tested a single injection of an experimental drug called BIVV001 in 6 adults with type 2N or type 3 von Willebrand disease, a rare bleeding disorder. The main goals were to see how the drug behaves in the body and whether it is safe. The study did not aim to cure the disease, but to gather information for future treatments.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Locations

Hemophilia Center of Western Pennsylvania_Investigational Site Number :8400001

Pittsburgh, Pennsylvania, 15213, United States
Investigational Site Number :2500001

Lille, 59037, France
Investigational Site Number :2500002

Nantes, 44093, France
University of Iowa_Investigational Site Number :8400002

Iowa City, Iowa, 52242, United States

Conditions

Explore the condition pages connected to this study.

VON WILLEBRAND'S DISEASE (VWD) VON WILLEBRAND'S DISEASE (VWD)